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Nrf2 Activator
Omaveloxolone for Friedreich's Ataxia
Phase 2
Waitlist Available
Research Sponsored by Biogen
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Have genetically confirmed Friedreich's ataxia
Be male or female and ≥16 years of age and ≤40 years of age
Must not have
Have uncontrolled diabetes (HbA1c >11.0%)
Have known active fungal, bacterial, and/or viral infection, including human immunodeficiency virus or hepatitis virus (B or C)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 weeks after participant receives the first dose in part 1
Summary
This trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.
Who is the study for?
This trial is for people aged 16-40 with genetically confirmed Friedreich's ataxia, who can swallow capsules and have a specific level of disease severity (modified FARS score between 20 and 80). Participants should be stable in their exercise routine and able to complete maximal exercise testing. They cannot join if they've used certain drugs recently, have significant heart disease or abnormal blood tests, uncontrolled diabetes, cognitive impairments, active infections, or substance abuse issues.
What is being tested?
The study examines omaveloxolone (RTA 408) capsules at various doses for treating Friedreich's ataxia. It has two parts: the first part tests safety across different doses; the second part assesses safety and effectiveness of a set dose (150 mg). An extension phase will look into long-term use. The drug aims to activate Nrf2 signaling impaired in this condition.
What are the potential side effects?
While not explicitly listed here, potential side effects may include reactions related to oxidative stress mitigation since omaveloxolone activates antioxidant pathways. These could involve changes in energy levels, gastrointestinal symptoms or possible liver enzyme alterations given its mechanism.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My condition is genetically confirmed Friedreich's ataxia.
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I am between 16 and 40 years old.
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I can swallow pills.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My diabetes is not under control (HbA1c >11.0%).
Select...
I do not have any active infections, including HIV or hepatitis.
Select...
I have previously participated in a trial with omaveloxolone.
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I have a history of serious heart disease.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline through 12 weeks after participant receives the first dose in part 1.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline through 12 weeks after participant receives the first dose in part 1.
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Change From Baseline in Peak Work (in Watts/kg) During Exercise Testing at Week 12 in Part 1
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 48 in Part 2
Secondary study objectives
Change in the Modified Friedreich's Ataxia Rating Scale (mFARS) at Week 12 in Part 1
Trial Design
10Treatment groups
Experimental Treatment
Placebo Group
Group I: Part 2 Omaveloxolone Capsules 150 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 150 mg administered orally once daily for 48 weeks
Group II: Part 1 Omaveloxolone Capsules 80 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 80 mg administered orally once daily for 12 weeks
Group III: Part 1 Omaveloxolone Capsules 40 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 40 mg administered orally once daily for 12 weeks
Group IV: Part 1 Omaveloxolone Capsules 300 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 300 mg administered orally once daily for 12 weeks
Group V: Part 1 Omaveloxolone Capsules 20 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 20 mg administered orally once daily for 12 weeks
Group VI: Part 1 Omaveloxolone Capsules 2.5 and 5 mgExperimental Treatment2 Interventions
omaveloxolone (RTA 408) Capsules, 2.5 mg administered orally one daily for 2 weeks, then 5 mg taken orally once daily for 10 weeks
Group VII: Part 1 Omaveloxolone Capsules 160 mgExperimental Treatment1 Intervention
Omaveloxolone (RTA 408) Capsules, 160 mg administered orally once daily for 12 weeks
Group VIII: Part 1 Omaveloxolone Capsules 10 mgExperimental Treatment1 Intervention
omaveloxolone (RTA 408) Capsules, 10 mg administered orally once daily for 12 weeks
Group IX: Part 1 Placebo CapsulesPlacebo Group1 Intervention
Placebo capsules administered orally once daily for 12 weeks
Group X: Part 2 Placebo CapsulesPlacebo Group1 Intervention
Placebo capsules administered orally once daily for 48 weeks
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Friedreich's Ataxia often focus on addressing oxidative stress and mitochondrial dysfunction, which are central to the disease's pathology. Omaveloxolone, for example, activates the Nrf2 pathway, leading to the induction of antioxidant genes that help mitigate oxidative damage.
This is crucial for Friedreich's Ataxia patients because their condition is characterized by impaired antioxidative defense mechanisms and mitochondrial iron overload, which contribute to cellular damage and disease progression. By enhancing the body's natural antioxidant defenses, treatments like Omaveloxolone aim to reduce oxidative stress and improve cellular function, potentially slowing disease progression and improving quality of life.
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Find a Location
Who is running the clinical trial?
BiogenLead Sponsor
645 Previous Clinical Trials
467,221 Total Patients Enrolled
2 Trials studying Friedreich Ataxia
148 Patients Enrolled for Friedreich Ataxia
Reata, a wholly owned subsidiary of BiogenLead Sponsor
49 Previous Clinical Trials
6,590 Total Patients Enrolled
3 Trials studying Friedreich Ataxia
340 Patients Enrolled for Friedreich Ataxia
Friedreich's Ataxia Research AllianceOTHER
18 Previous Clinical Trials
6,088 Total Patients Enrolled
18 Trials studying Friedreich Ataxia
6,088 Patients Enrolled for Friedreich Ataxia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My condition is genetically confirmed Friedreich's ataxia.I am between 16 and 40 years old.I have not taken and will not take certain drugs that affect liver enzymes or drug transporters during the study.My diabetes is not under control (HbA1c >11.0%).I have difficulty understanding or remembering things, which may affect my ability to follow study procedures.You can complete a very challenging exercise test.I do not have any active infections, including HIV or hepatitis.You have important problems with your blood or body chemistry, like high levels of certain enzymes.Your B-type natriuretic peptide level is higher than 200 pg/mL.I have previously participated in a trial with omaveloxolone.Your modified FARS score is between 20 and 80.You are currently using drugs or drinking alcohol excessively.I have a history of serious heart disease.I can swallow pills.
Research Study Groups:
This trial has the following groups:- Group 1: Part 1 Omaveloxolone Capsules 10 mg
- Group 2: Part 1 Omaveloxolone Capsules 2.5 and 5 mg
- Group 3: Part 1 Omaveloxolone Capsules 20 mg
- Group 4: Part 1 Omaveloxolone Capsules 40 mg
- Group 5: Part 1 Omaveloxolone Capsules 80 mg
- Group 6: Part 1 Omaveloxolone Capsules 160 mg
- Group 7: Part 1 Omaveloxolone Capsules 300 mg
- Group 8: Part 1 Placebo Capsules
- Group 9: Part 2 Placebo Capsules
- Group 10: Part 2 Omaveloxolone Capsules 150 mg
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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